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Positive Preclinical Profile Of RG-101, A GalNAc-conjugated Anti-miR Targeting MicroRNA-122, Supports Clinical Development For The Treatment Of HCV

LA JOLLA, Calif., Nov. 4, 2013 /PRNewswire/ -- Regulus Therapeutics Inc. (NASDAQ: RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, today announced that data demonstrating the positive preclinical profile of RG-101 will be presented in a late-breaking poster session at the 64th Annual Meeting of the American Association for the Study of Liver Disease (AASLD) being held in Washington, D.C. on Monday, November 4, 2013 from 8:00 a.m. Eastern Standard Time (EST) to 5:30 p.m. EST. The poster is available on the Company's website at        

"RG-101 utilizes a unique mechanism of action by targeting microRNA-122, a liver-specific host factor for stability, replication and translation of HCV.  We believe that therapies that target host-encoded factors essential for HCV replication may act as attractive combination agents because they may demonstrate activity across all HCV genotypes and may have a high barrier to resistance," said Neil W. Gibson, Ph.D., Chief Scientific Officer of Regulus.  "We continue to be encouraged by the preclinical data seen to date and believe that RG-101 has the potential to be a best-in-class host factor agent.  In the near term, we expect to file our application with regulatory authorities and look forward to commencing clinical trials in man in early 2014."

Late-Breaking Poster on RG-101 – Monday, November 4, 2013, 8:00 a.m. EST5:30 p.m. EST

- RG-101, a GalNAc-conjugated anti-miR Employing a Unique Mechanism of Action by Targeting Host Factor MicroRNA-122 (miR-122), Demonstrates Potent Activity and Reduction of HCV in Preclinical Studies- 

In the late-breaking poster, Regulus will present positive data from completed preclinical studies evaluating RG-101 for in vitro and in vivo potency, pharmacokinetic/pharmacodynamics, toxicology and safety pharmacology and inhibition of HCV replication. 

Potency and Pharmacokinetics/Pharmacodynamics of RG-101

Pharmacologic potency of RG-101 was significantly enhanced by approximately 20 fold in vivo in both mice and non-human primates, relative to the unconjugated oligonucleotide of RG-101 (RG-101-N).  RG-101 is rapidly taken up in the liver and metabolized to the active oligonucleotide, which has an approximately 14 day tissue half-life.  Additionally, potency as measured by AldoA (a miR-122 target gene) de-repression in the liver is achieved at significantly lower liver concentrations of oligonucleotide following efficacious doses of RG-101 of 1mg/kg-10mg/kg, compared to similar doses of RG-101-N.  In addition to the potency studies, Regulus tested the efficacy of RG-101 to reduce HCV viral load titer in a human chimeric liver mouse model infected with HCV, as these mice are estimated to contain greater than 80 percent human hepatocytes.  Genotype 1a HCV infected mice were subcutaneously administered a single dose of RG-101 at 3 mg/kg, 10mg/kg or 30 mg/kg and monitored for up to 36 days.  Up to a 2 log reduction in HCV viral load titer was observed, which is similar to that observed for oral direct-acting antivirals as monotherapy in this mouse model.  Additionally, the duration of action observed for RG-101 supports the potential for a once-a-month dosing regimen.

Regulus believes these preclinical data will assist the Company in determining the most efficacious dose of RG-101, including optimal dosing frequency, for clinical studies in man. 

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